In a promising finding for research into Huntington’s disease (HD) — the devastating inherited neurodegenerative disorder — a team of UC Davis investigators has identified for the first time a way to use human mesenchymal stem cells (MSCs) to deliver the key brain protein growth factor that is dramatically diminished by Huntington’s disease. The discovery could be the precursor to developing a successful therapy to treat those who suffer from the disease.

uniQure N.V. a leader in human gene therapy, today announced the publication of preclinical data supporting its proprietary Huntington’s disease gene therapy program, AMT-130. Findings published in the current issue of the peer-reviewed journal Molecular Therapy-Nucleic Acids(www.nature.com/mtna/journal/v5/n3/index.html) provide preclinical proof of concept for uniQure’s AMT-130 program and demonstrate the potential of a one-time administration of AAV5-delivered gene therapy into the central nervous system